Efficacy and Safety of Ruxolitinib in Neuromyelitis Optica Spectrum Disorders

Neuromyelitis Optica Spectrum Disorders (NMOSD) is associated with a pathological humoral immune response against the aquaporin-4(AQP-4) water channel. Rucotinib is an oral inhibitor of JAK1 and JAK2 tyrosine kinases. It may benefit some patients with NMOSD due to the important role of JAK/STAT signaling pathway in the pathogenesis of NMOSD. Clincial trials may be needed to observe its efficacy and safety.

Study Overview

• Status: Withdrawn
• Conditions: Neuromyelitis Optica Spectrum Disorder Relapse
• Intervention / Treatment: Drug: Ruxolitinib

• Study Type: Interventional
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Tianjin
    • Tianjin, Tianjin, China, 300052
      • Tianjin Medical University General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Male or female patients ≥ 18 years old; Diagnosis of NMO or NMO spectrum disorder according to the 2015 International diagnostic criteria for neuromyelitis optic; Clinical evidence of at least 2 relapses in last 12 months or 3 relapses in the last 24 months; EDSS <= 6.0; Rituximab should be used for at least 3 months if the condition is stable; Able and willing to give written informed consent and comply with the requirements of the study protocol.

Exclusion Criteria:

Current evidence or known history of clinically significant infection (Herpes simplex virus, varicella-zoster virus, cytomegalovirus, Epstein-Barr virus, human immunodeficiency virus, Hepatitis viruses, Syphilis, etc); Participation in another interventional trial within the last 3 months; Patients taking oral immunosuppressants such as azathioprine; Tumor disease currently or within last 5 years; Pregnant, breastfeeding, or child-bearing potential during the course of the study; Clinically relevant anemia, thrombocytopenia and dysfunction of the heart, liver, kidney or bone marrow.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Ruxolitinib; Treatment with ruxolitinib will be initiated in an initial dose regimen of 5-10 mg twice daily. Two months later, the dose of ruxolitinib will be increased to 10-15 mg twice daily.	Drug: Ruxolitinib; Treatment with ruxolitinib will be initiated in an initial dose regimen of 5-10 mg twice daily. Two months later, the dose of ruxolitinib will be increased to 10-15 mg twice daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
time to the first protocol-defined relapse	An acute attack was defined as a new neurological worsening lasting for at least 24 hours and occurring more than 30 days after the previous attack.	From baseline to one year after.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Worsening in EDSS	The Expanded Disability Status Scale (EDSS) is a rating system that is frequently used for classifying and standardizing the severity and progression. EDSS ranges from 0 to 10.	Worsening from baseline in EDSS to 52 weeks
Incidence of treatment-emergent adverse events [safety and tolerability]	Adverse events related to ruxolitinib are recorded	From baseline to 52 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Counts of peripheral blood B cell subsets	Compare peripheral blood plasma cells before and one year after initial intervention.	From baseline to 52 weeks
Number of New, and/or Enlarging T2 Hyperintense Lesions as Detected by Optic nerve,brain and spinal cord Magnetic Resonance Imaging (MRI)	The total number of new and/or enlarging T2 lesions for all participants was calculated as the sum of the individual number of lesions at Weeks 12, 24, and 52.	From baseline to 52 weeks
Determination of serum AQP4 antibodies	Compare serum AQP4-ab titers before and one year after initial intervention.	From baseline to 52 weeks

Collaborators and Investigators

• Sponsor: Tianjin Medical University General Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2024
• Primary Completion (Estimated): February 1, 2026
• Study Completion (Estimated): August 1, 2026

Study Registration Dates

• First Submitted: March 8, 2023
• First Submitted That Met QC Criteria: June 10, 2023
• First Posted (Estimated): June 19, 2023

Study Record Updates

• Last Update Posted (Estimated): June 19, 2023
• Last Update Submitted That Met QC Criteria: June 10, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Eye Diseases; Optic Nerve Diseases; Cranial Nerve Diseases; Myelitis, Transverse; Optic Neuritis; Neuromyelitis Optica
• Other Study ID Numbers: IRB2022-YX-221-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No